Ionis Enters Global Licensing Deal with Recordati for Alexander disease ASO

Rare Daily Staff

Ionis Pharmaceuticals has struck a global licensing deal with Italy-based Recordati to bring its experimental therapy zilganersen to people with the rare and often fatal neurological condition Alexander disease outside the United States.

Under the agreement, Ionis will receive a $30 million upfront payment from Recordati and could earn additional milestone payments and royalties on future sales. The financial terms reflect the high unmet need and small patient population typical of ultra-rare diseases.

Ionis will continue to lead global development and retain full commercial rights in the United States, where the drug is currently under U.S. Food and Drug Administration review, with a decision expected by September 22.

The deal underscores growing industry interest in ultra-rare neurological disorders—particularly those with well-defined genetic causes that can be targeted with precision therapies such as antisense drugs.

For Ionis, the drug also represents a strategic milestone. If approved, zilganersen would be the company’s first independently commercialized neurology product in the United States, building on its history of RNA-targeted therapies, including Spinraza for spinal muscular atrophy.

Recordati will take responsibility for regulatory filings, market access, and commercialization in all other countries.

Zilganersen is an antisense oligonucleotide designed to reduce levels of glial fibrillary acidic protein (GFAP), a protein that accumulates abnormally in Alexander disease due to mutations in the GFAP gene. This buildup disrupts astrocytes—support cells in the brain—leading to progressive neurological decline. Patients often experience worsening motor function, loss of independence, and difficulty swallowing and breathing. The disease typically leads to death within 14 to 25 years after symptom onset.

In a pivotal study, patients aged five and older who received zilganersen showed statistically significant stabilization in walking ability, measured by the 10-meter walk test, compared with untreated patients at 61 weeks. Additional measures reported by clinicians and caregivers also suggested broader functional benefits. The drug appeared generally well tolerated, with fewer serious adverse events reported in treated patients than in the control group.